The first phase of the U.K. synthetic human genome project has successfully completed, realizing key steps in chromosome synthesis. The work has demonstrated a multistep method for transfecting mouse stem cells with native human chromosomes, where they are stably maintained and can be manipulated to replace native DNA with synthetic DNA. The engineered chromosomes can then be transferred into a human cell in place of the native chromosomes.
The first phase of the U.K. synthetic human genome project has successfully completed, realizing key steps in chromosome synthesis. The work has demonstrated a multistep method for transfecting mouse stem cells with native human chromosomes, where they are stably maintained and can be manipulated to replace native DNA with synthetic DNA. The engineered chromosomes can then be transferred into a human cell in place of the native chromosomes.
Laigo Bio BV has raised €11.5 million (US$13.7 million) in a seed round for the further development of a new approach to inducing targeted protein degradation by E3 ubiquitin ligases.
Neurimmune AG has expanded its transthyretin amyloid cardiomyopathy (ATTR-CM) collaboration with Alexion, Astrazeneca Rare Disease by entering into an exclusive global collaboration and license agreement to develop NI-009, a fibril depleter for light chain (AL) amyloidosis.
CSPC Pharmaceutical Group Ltd. has obtained IND clearance from the FDA to conduct clinical trials in the U.S. with SYH-2069 for weight management in individuals with obesity or overweight and at least one weight-related comorbidity.
Crescent Biopharma Inc. has announced a new partnership with Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd. and reported progress across its pipeline, with three distinct programs set to enter the clinic next year.
A recently published study, conducted by researchers from Jinan University and collaborating institutions, aimed to investigate the mechanisms of endometriosis-driven carcinogenesis.
Wigen Biomedicine Technology (Shanghai) Co. Ltd. has disclosed phosphatidylinositol 3-kinase α (PI3Kα) inhibitors reported to be useful for the treatment of solid cancers and hematological cancers.
Avencell Therapeutics Inc. has received clinical trial clearances from the FDA and EMA to conduct a phase I/II trial (Quadvance) of AVC-203 for the treatment of relapsed or refractory B-cell malignancies.
At the Clinical Trials on Alzheimer’s Disease 2025 meeting, a panel of experts discussed the need for developing combination therapies for the complex diseases that result in Alzheimer's disease and other dementias.
Abilita Therapeutics Inc. has developed a novel approach targeting CCR8 – ABT-863 – that works as a potent inverse agonist to block CCL1-dependent and basal receptor signaling.
LRRK2 plays a key role in the biology of multiple neurodegenerative disorders, including Parkinson’s disease (PD) and progressive supranuclear palsy (PSP), where mutations or altered activity are associated with impaired cellular signaling and neuronal decline.
Previous findings have shown that the bisteric mTORC1 inhibitor Rapalink-1 exerts superior efficacy than the parent inhibitors rapamycin and sapanisertib in orthotopic xenograft models of glioblastoma. The bitopic clinical derivative of this compound is RMC-5552 from Revolution Medicines Inc.,...
Vycellix Inc. has successfully completed preclinical development for its universal cell engineering platform (VY-UC) and will now seek clinical trial clearance in Sweden to begin a phase I study of VNK-101, an allogeneic natural killer (NK) cell therapy engineered with VY-UC for relapsed or...
2025 has been the most challenging year in the efforts to fight HIV since at least the advent of antiretroviral therapy. In a report on “Overcoming disruption, transforming the AIDS response,” released last week ahead of World AIDS Day on Dec. 1, the Joint United Nations Program on HIV/AIDS...
Researchers from the Medical School of Nanjing University hypothesized that in ulcerative colitis, the gut-resident macrophages may be compromised, leading to impaired integrity of the epithelial barrier. “From a basic science standpoint, our work uncovers a novel etiology of ulcerative colitis....
Researchers at Shandong Second Medical University have developed a dimethyl quaternary ammonium derivative of harmane that shows strong potential for attacking the bacterium through diverse mechanisms.
Arrakis Therapeutics Inc. has announced the presentation of preclinical data demonstrating the progress of its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1).
Lynk Pharmaceuticals Co. Ltd. has received IND clearance from the FDA for LNK-01006, an allosteric TYK2 inhibitor with potential to treat neurodegenerative diseases.
Vectory Therapeutics BV has obtained IND clearance from the FDA for a phase I/II trial (PIONEER-ALS) of VTX-002, a first-in-class vectorized antibody targeting TDP-43 pathology in amyotrophic lateral sclerosis (ALS). VTX-002 delivers an engineered antibody designed to selectively target toxic...
Latus Bio Inc. has reported IND clearance by the FDA for LTS-101, a gene therapy candidate to treat the CNS manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. The FDA has also granted orphan drug, rare pediatric disease and fast track designations to LTS-101.
Junevity Inc. has raised $10 million in new funding, bringing its seed financing to a total of $20 million. The new funding will enable Junevity to advance JUN-01, its lead siRNA program for type 2 diabetes and obesity, through IND-enabling and initial clinical studies.
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA)...
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.
Despite the formidable challenges for developing precision psychiatry, the approach is notching its first successes in the preclinical and even some clinical settings. Many individual studies as well as large projects like the Psychiatric Ratings using Intermediate Markers (PRISM) studies and the...
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH)...
The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of...
Samjin Pharmaceutical Co. Ltd. has disclosed inhibitors of 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) and/or estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) and/or HSD17B2 (17β-HSD2). As such, they are believed to be...
Imhotex Ltd. has disclosed desmuramylpeptide (DMP) analogues of muramyl dipeptide (MDP) acting as nucleotide-binding oligomerization domain-containing protein 2 (NOD2) activators reported to be useful for the treatment of Crohn’s disease.
Numab Therapeutics AG and Kaken Pharmaceutical Co. Ltd. have entered into a strategic licensing and co-development agreement for NM-81, a multi-specific antibody for the treatment of inflammatory bowel disease (IBD).
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